Gene Therapy Helps Treat Chronic Traumatic Encephalopathy

Gene therapy was found to help treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study, which demonstrated the effectiveness of direct delivery of gene therapy into the brain of a mouse model of //CTE, is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the full-text article free on the Human Gene Therapy website through February 3, 2020.

Ronald Crystal and colleagues from Weill Cornell Medical College, New York, NY, coauthored the article entitled "Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy." Inflammation results in the accumulation of hyperphosphorylated forms of Tau protein (pTau).

Crystal et al. developed an adeno-associated virus (AAV) vector to deliver an anti-pTau antibody to the (CNS). They showed that direct delivery of the AAVrh.10anti-pTau directly into the hippocampus of brain-injured mice was associated with a significant reduction in pTau levels across the CNS. They propose that doses could be scaled up and this strategy could be effective in humans as well.

"CTE is much more prevalent than was initially realized, and there is currently no therapy available," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "This new work from the Crystal laboratory is potentially ground-breaking as a means to remove the offending Tau phoshoprotein."

Source-Eurekalert